Clinical Trial

Muscular Dystrophy

RESTEM, LLC has partnered with University of Florida in this Phase I clinical trial treating children with Duchenne Muscular Dystrophy (DMD). This FDA-cleared, double-blind, placebo-controlled clinical study will administer our umbilical cord lining stem cells (ULSCs) to children 6 or more years of age suffering from DMD. The objective of this study is to slow the progression of disease and improve the quality of life.

Duchenne Muscular Dystrophy is a rapidly progressive form of Muscular Dystrophy primarily affecting young males. Most DMD patients experience early signs of muscle weakness by the age of 4 and are usually confined to a wheelchair by the age of 12. DMD not only affects mobility, but also causes heart muscle abnormalities. Weakening of the cardiac muscle, known as cardiomyopathy, causes heart failure with progressive breathing difficulty and rapid deterioration in the patient’s quality of life. In most cases, Duchenne is fatal by the age of 30.

We aim to improve the quality of life for those suffering from this crippling disorder and help these children live a more carefree childhood. If this Phase I trial is successful, a Phase II study is planned that will evaluate this therapy in a larger patient population, with the overarching goal of bringing this next-generation therapy to routine clinical care.

To learn more about this trial, visit: clinical trial information.pdf (

To read the Press Release of this trial, visit: RESTEM and JAR of Hope Foundation Announce Strategic Partnership and Clinical Trial Launch of Next-Generation Cell Therapy for Patients with Duchenne Muscular Dystrophy (

If you are interested in learning more or getting involved, please contact us